MissionEB

• Research type

  Research Study

• Full title

  Mesenchymal Intravenous Stromal cell Infusions in children with recessive dystrophic Epidermolysis Bullosa

• IRAS ID

  281748

• Contact name

  Anna Martinez

• Contact email

  anna.martinez@gosh.nhs.uk

• Sponsor organisation

  Great Ormond Street Hospital NHS Foundation Trust

• Eudract number

  2020-005049-18

• ISRCTN Number

  ISRCTN14409785

• Duration of Study in the UK

  4 years, 4 months, 31 days

• Research summary

  Background:
  Recessive Dystrophic Epidermolysis Bullosa (RDEB) is an inherited skin blistering disease. RDEB is a severe form of epidermolysis bullosa (EB) caused by loss-of-function mutations in type VII collagen gene (COL7A1) leading to skin blistering following minor mechanical trauma. There is no effective treatment for RDEB and management is mainly supportive. The use of mesenchymal stromal cells (MSCs) in children with RDEB has been studied before and was found to improve wound quality, reduce skin itching and pain without significant side effects.
  Objectives:
  The aim of this study is to assess the safety of repeated infusions of MSCs in children with RDEB. The study will also assess the efficacy of MSCs in improving the disease severity, quality of life and symptoms (e.g. pain and itch) including the cost-effectiveness compared to the usual care over the 15-month period in children with severe RDEB.
  Method:
  This is a prospective, randomised, placebo-controlled, double-blinded, 15-month cross-over trial with a 12-month open label continued treatment follow-on study. A total of 36 eligible subjects will be recruited at both clinical sites (Great Ormond Street Hospital and Birmingham Children’s Hospital). An internal phase 1 study to assess the safety of the MSCs will be carried over the first 4 months. Each subject will be randomised and enrol through a 2-period, cross-over study to receive repeated infusions of either MSCs followed by placebo or placebo followed by MSCs in order to evaluate efficacy. The patients will be assessed by using EB severity scores, e.g. EBDASI and iscorEB, clinical photography, pain and itch assessment, quality of life questionnaires and routine safety blood tests.

• REC name

  North East - York Research Ethics Committee

• REC reference

  21/NE/0016

• Date of REC Opinion

  29 Mar 2021

• REC opinion

  Further Information Favourable Opinion